Soon available in:

The study for pregnant women

of an XLHED-affected boy

The EDELIFE study

If you are a pregnant woman (aged 18 or over), with a confirmed genetic diagnosis of XLHED, expecting a boy and if your unborn son might be affected by XLHED, this section presents the 3 different periods of the study for you and your son.

If you choose to participate in this study, you will be asked to come to visits to the clinical site for each study period.

It is also recommended that you give birth at the clinical site.

Altogether, there will be 14 visits over 5 years.

The EDELIFE study will consist of 3 periods:
Screening, Treatment and Follow-up.

Below is a graphical representation of the study, its different phases, the different visits and how long they take:

Visit (V)
14 visits over 5+ years
4 while pregnant
1 at delivery
9 afterwards
Pregnant Woman

Screening Period

Expected visit length :
1⁄2 day to 1 day on site

Treatment Period

Expected visit length : 
2 days (overnight stay) on site for each visit
Child: from birth to 5 years old
Mother: up to 1 month after delivery

Follow-up Period

Expected visit length :
1⁄2 day to 1 day on site for each visit

Follow up Period

Expected visit length :
1⁄2 day to 1 day on site for each visit

Follow up Period

Expected visit length :
1⁄2 day to 1 day on site for each visit
1

Screening Period

2

Treatment Period

3

Follow-up Period

The Screening period

The screening consists of 1 visit to the clinical site. This visit is anticipated to take half a day to a full day and must take place between pregnancy week 19 and the end of week 24.

This visit will confirm that you are carrying a boy. The study team will check if your unborn son is affected or not by XLHED using a non-invasive ultrasound scan.
The doctors will take a blood sample and conduct a series of other, non-invasive medical tests on you, to ascertain whether you and your unborn son can participate in the study (i.e. decide whether you are eligible).

Illustration of the procedures that you will undergo at the Screening visit

If you are eligible and choose to participate in the study, you will come back later for treatment of your unborn son.

If you do not choose to participate or if you are not eligible, you will not be asked to come back to the clinical center for the study.

Please be reassured that your medical care will not be affected by this decision.
Please take all the time you need to reflect before the Screening visit. If it helps, discuss the study with your family, friends or medical team (midwife, General/family Doctor, etc) before giving your consent or deciding not to participate.

At the Screening visit, you will get a chance to discuss in person with the study doctor. It might be a good idea that you prepare and have ready some written questions.

The Treatment period

Treatment for your unborn child will take place at the clinical site over 3 visits, approximately 3 weeks apart, at pregnancy weeks 26, 28-29 and 31-32.

At each visit, you will receive one ER004 injection. Each visit will require you to attend the site for 2 days. Before each injection, a health check for both you and your baby will be conducted, to ensure all is well and you can receive the treatment. After the injection, you will rest and be required to spend the night on site for observation.

At the first treatment visit, we will ask you to have an MRI scan. This is to assess the tooth development of your baby before ER004 treatment.

The procedure is not generally considered to be painful, but the machine makes a loud noise. The procedure should not last more than 90 minutes.

As part of the first treatment visit, we will ask to interview you. This interview will be conducted remotely and you will not need to go to the clinical site for this. This interview will be conducted by a trained interviewer, the aim of which is to hear your viewpoints, experiences, feelings and opinions on XLHED and the ER004 treatment. This interview is recorded (audio only) and should not last longer than 25 minutes.

No treatment will take place after your baby is born.

Illustration of the procedures that you will undergo at a Treatment visit

The Follow-up period

The follow-up period starts with the birth of your child and will last until your son is 5 years old.

After delivery, you will be asked to come to the clinical site for 9 more visits, with your son.

He will be
1 month
3 months
6 months
12 months
18 months
2 years
3 years
4 years
5 years

Medical examinations of your son will allow us to evaluate if the treatment has worked and how safe it is.

Each visit will last between a half and a full day. Slightly different tests will be conducted at different visits.

At these follow-up visits, your son will undergo many tests to check his XLHED symptoms and make sure he is doing well. These tests are all non-invasive.

The doctors will take a blood sample from your son on 3 separate follow up visits. Analysing your son’s blood will show very important information on how well your son has coped with the treatment.

Altogether, the blood volume taken over these 3 visits will represent less than a teaspoon (5 mL). Blood will be drawn by a trained professional who is used to dealing with small infants.

One MRI assessment is planned between the ages of 3 and 6 months. For this, your son will not require sedation. Throughout this procedure, you will remain next to your son. The procedure is not generally considered to be painful, but the machine makes a loud noise. The whole procedure should take no more than 90 minutes.
One X-ray will be taken between 36 and 60 months of age.

It is important to assess the outcome of the treatment on your son’s tooth growth and his teeth will be examined at each visit.

At some visits, you will be asked to fill in questionnaires about your and your son’s quality of life. This should take no longer than 40 minutes.

As mentioned in the treatment period, we will ask to interview you again.

These interviews will be conducted in the same way as during the treatment period. Your child does not need to be present.

This interview is recorded (audio only) and should take no longer than 40 minutes.

You will also undergo some tests, but only at delivery and at the follow-up visit immediately after delivery, to ensure your health has not been affected by your son’s treatment.

Illustration of the procedures that you will undergo at a Follow-up visit

What if I don’t want to participate anymore?

Please remember that you have the right to decide, at any point during the study, not to participate any longer, even if you had initially chosen to participate.This decision will not affect the medical care you receive in any way.
Copyright © 2021 – 2022 • Groupe Pierre Fabre All Rights Reserved
crosschevron-leftchevron-right